Sign in or register to access exclusive content on this site
REGISTER

Home | Neuromuscular | Duchenne muscular dystrophy (DMD) | Publications | Publication: Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment (NSAA)

Publication: Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment (NSAA)

This research article, published in Muscle and Nerve, describes investigations into a well-developed analytical procedure for evaluating longitudinal treatment effects using the North Star Ambulatory Assessment (NSAA) data for illustration

  • The analytical approach for evaluating the longitudinal treatment effects of ataluren vs. placebo and deflazacort vs. prednisone/prednisolone among placebo-treated patients in Duchenne muscular dystrophy (DMD) is described
  • The comparison between the NSAA approach, which comprises 17 scorable items/outcomes to measure changes in motor function and a fixed-time analysis, is reviewed
  • Potential benefits of evaluating disease burden by assessing the totality of treatment effects using multiple outcomes measures per patient, as per the NSAA, are addressed
  • The feasibility of using this approach to shape future clinical trials in place of a fixed-time analysis is discussed

▼ Ataluren is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC Therapeutics at pharmacovigilance@ptcbio.com.

Registration conditions differ internationally; always consult local prescribing information and/or Summary of Product Characteristics before prescribing any products.

MED-ALL-ATLN-2500006 | September 2025


PTC Therapeutics is the Marketing Authorization Holder for deflazacort in the US only. Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The US Prescribing Information for deflazacort is available here. Please refer to your local country guidance for more information.

For medical information, product complaints, or to report an adverse event, please call 1-866-562-4620 or email at usmedinfo@ptcbio.com. You may also report adverse events directly to FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

MED-ALL-DFZ-2500002 | March 2025

McDonald CM, Wei LJ, Flanigan KM, et al. Muscle Nerve. 2021;64(5):614–619

Once registered, you will be able to view the abstract and be provided the DOI and PubMed links for this publication.

Thank you!

Already registered?

Registration
* indicates a required field
  • This Website is intended only for healthcare professionals (“Website“), is operated by PTC Therapeutics. (together with its affiliates “PTC”). In order to access the restricted content on this Website:

    *
    *
  • PTC will delete immediately the information provided if we cannot confirm you are a healthcare professional.
    Otherwise, in case of successful verification, PTC will retain the information provided for a period of two (2) years following your registration (i) only for the purposes of providing you access to this Website; and/or (ii) in case you signed up to receive PTC updates.

Providing your personal data and your consent is voluntary, and you have the right to withdraw your consent at any time, without giving any reasons. Please consult our Privacy Statement for more information on the processing of your personal data.
For any inquiry relating to the processing of your personal data or if you want to exercise your privacy related rights or withdraw your consent, you can contact PTC’s Privacy Office at: Dataprivacy@ptcbio.com.

You can unsubscribe from updates at any time when receiving an email from PTC.

MED-ALL-DMD-2300008 | June 2025

Welcome to MEDhub

A website for healthcare professionals, provided by PTC Therapeutics

This educational website provides you with the latest scientific data and expert insights on the diagnosis and management of a range of rare diseases across the neuromuscular system, central nervous system, and other areas

This website may include scientific information about investigational and unlicensed products not approved for use within your country. Please refer to local regulatory guidance for country-specific information

© 2025 PTC Therapeutics. All rights reserved | MED-ALL-CORP-2200029 | June 2025

This content is protected.

To view it please enter your email address below:

Register here to access the content on the site

MED-ALL-CORP-2200029 | June 2025

COMING SOON

The content you are trying to access is not currently available.

We will be updating the MEDhub site regularly to provide you with up-to-date, insightful expert-led content. To be notified when new additions become available, register now.